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Placebo matching Pegcetacoplan
This is a placebo control arm matching the active drug pegcetacoplan, which is a complement C3 inhibitor that blocks the alternative complement pathway.
This is a placebo control arm matching the active drug pegcetacoplan, which is a complement C3 inhibitor that blocks the alternative complement pathway. Used for Paroxysmal nocturnal hemoglobinuria (PNH), C3 glomerulopathy, Post-transplant thrombotic microangiopathy.
At a glance
| Generic name | Placebo matching Pegcetacoplan |
|---|---|
| Sponsor | Swedish Orphan Biovitrum |
| Drug class | Complement C3 inhibitor (aptamer) |
| Target | Complement C3 |
| Modality | Small molecule |
| Therapeutic area | Immunology / Rare Disease |
| Phase | Phase 3 |
Mechanism of action
Pegcetacoplan is a pegylated aptamer that binds and inhibits complement component C3, preventing its cleavage and activation of the alternative complement pathway. By reducing complement-mediated inflammation and cell destruction, it aims to treat complement-driven diseases. As a placebo matching formulation, this control contains no active drug and is used for blinded comparison in clinical trials.
Approved indications
- Paroxysmal nocturnal hemoglobinuria (PNH)
- C3 glomerulopathy
- Post-transplant thrombotic microangiopathy
Common side effects
- Injection site reactions
- Headache
- Nausea
Key clinical trials
Primary sources
Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.
| Source | Used for |
|---|---|
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |